Tiny engineered therapeutic delivery system safely solves genetic problems in mice

Friday, August 21, 2020
(Funded by the National Institutes of Health)

Scientists have demonstrated that lipid-based nanoparticles carrying two sets of protein-making instructions have the potential to function as therapies for two genetic disorders. In one experiment, the payload-containing nanoparticles prompted the production of the missing clotting protein in mice that were models for hemophilia. In another test, the nanoparticles' cargo reduced the activation level of a gene that, when overactive, interferes with clearance of cholesterol from the bloodstream.